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Relapsed Acute Myeloid Leukemia (AML)Phase 1NCT06846606

Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS

A Phase 1 study testing AUTX-703 in people with Relapsed Acute Myeloid Leukemia (AML) and Refractory Acute Myeloid Leukemia (AML), run by Auron Therapeutics, Inc. at 9 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 administered orally in subjects with advanced hematologic malignancies. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
AUTX-703
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
69 participants
Where
9 sitesUnited States
Study dates
1 May 2025to Jun 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participant must be ≥18 years of age
  • Participant must have confirmed diagnosis as follows:
  • R/R AML and has not achieved adequate response to, or cannot tolerate, all approved therapies known to be active for treatment of their disease OR R/R MDS with over 10% blasts in the bone marrow and has not achieved an adequate response to at least 4 cycles of a hypomethylating agent (HMA)- containing regimen or other treatment known to be active for their disease OR R/R AML or R/R MDS that has relapsed after a hematopoietic stem cell transplant (HSCT)
  • Participant must be willing and able to comply with scheduled study visits and treatment plans.
  • Participant must be willing to undergo all study procedures unless contraindicated due to medical risk.
  • Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-1
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  • Participant must have adequate hepatic function
  • Participant must have adequate renal function
  • Participant must have adequate cardiovascular function
  • Participant must have a white blood cell (WBC) count ≤20 × 10⁹/L (with stable hydroxyurea use allowed)
  • Participant must meet timing requirements with respect to prior therapy and surgery
  • Participant must agree to use effective contraception during the study and for the required post-treatment period: Males: Use condoms (even if vasectomized) during the study and for 90 days post-treatment. Females of childbearing potential: Use a combination of 1 highly effective and 1 effective method of contraception during the study and for 180 days post-treatment.

Who cannot join

  • Participant is unable to provide informed consent and/or to follow protocol requirements.
  • Participant has undergone chimeric antigen receptor T cell therapy or HSCT within 60 days of the first dose of study treatment or has active clinically significant graft-versus-host disease (GVHD)
  • Participant has another malignancy that may interfere with diagnosis and treatment of R/R AML or R/R MDS.
  • Participant has an active severe infection that requires anti-infective therapy or has an unexplained temperature of >38.5°C during screening visits or on their first day of study treatment.
  • Participant has a known sensitivity to AUTX-703 or any of its components.
  • Participant is taking systemic strong CYP3A4 inhibitors or inducers within 14 days of the first dose of study treatment.
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  • Participant who are taking proton pump inhibitors should be switched to another acid-reducing agent such as an antacid or H2 blocker
  • Participant is taking P-gp and breast cancer resistance protein (BCRP) inhibitors or inducers within 14 days of first dose of study treatment.
  • Participant has active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections with detectable viral load
  • Participant has experienced AIDS related illness within the past 6 months or have detectable HIV viral load.
  • Participant has an uncontrolled intercurrent illness
  • Participant has active Class III or IV cardiovascular disease within 6 months prior to the start of study treatment
  • Participant is unable to tolerate the administration of oral medication or has GI dysfunction that would preclude adequate absorption, distribution, metabolism, or excretion of an oral medication
  • Participant is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment
  • Have a history of interstitial lung disease or pneumonitis.

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Auron Clinical Trials contact

617-514-2356

clinicaltrials@aurontx.com

9 sites in 1 country

United States9 sites, 9 recruiting

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203

    Recruiting

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27514

    Recruiting

  • Ohio State University, The James Comprehensive Cancer

    Columbus, Ohio, 43210

    Recruiting

  • City of Hope National Medical Center

    Duarte, California, 91010

    Recruiting

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

    Recruiting

  • Sarah Cannon Center for Blood Cancer at TriStar Centennia

    Nashville, Tennessee, 37203

    Recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10021

    Recruiting

  • UPENN Perelman Center for Advanced Medicine

    Philadelphia, Pennsylvania, 19104

    Recruiting

  • H Lee Moffitt Cancer Center and Research Institute

    Tampa, Florida, 33612

    Recruiting

Other trials in United States

About this study

Official title: A Phase 1 Study of AUTX-703 in Participants With Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 administered orally in subjects with advanced hematologic malignancies.

Read the detailed description

This is a first-in-human, Phase 1, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703, an orally bioavailable lysine acetyltransferase 2A (KAT2A) and lysine acetyltransferase 2B (KAT2B) degrader, in participants with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The study consists of two parts: Part A (Dose Escalation) to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D), and Part B (Dose Optimization) to further evaluate safety, PK, PD and efficacy at selected dosages.

Being tested
AUTX-703
Keywords
AUTX-703, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed/Refractory AML, Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, KAT2A/B degrader, Myelodysplastic Syndromes
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AUTX-703Drug

    AUTX-703 administered orally

Study groups in NCT06846606
Study groupRoleWhat they receive
Dose Escalation - Part AExperimentalParticipants will receive escalating dosages of AUTX-703 orally in tablet form once, twice or three times weekly to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D).
Receives: Drug: AUTX-703
Dose Optimization - Part B, Dosage 1ExperimentalParticipants will receive AUTX-703 at the first selected dosage determined from Part A, administered orally in tablet form either once, twice or three times weekly to further evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity at this specified dose.
Receives: Drug: AUTX-703
Dose Optimization - Part B, Dosage 2ExperimentalParticipants will receive AUTX-703 at the second selected dosage determined from Part A, administered orally in tablet form either once, twice or three times weekly to further evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity at this specified dose.
Receives: Drug: AUTX-703

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Adverse Events (AEs), Dose-Limiting Toxicities (DLTs), and Serious Adverse Events (SAEs)

    To assess the safety and tolerability of AUTX-703 by evaluating the incidence and severity of AEs, DLTs, SAEs, and AEs leading to treatment discontinuation.

    Measured: From the first dose through 28 days after the last dose of study drug.

Secondary outcomes (26)

  1. To Identify the Recommended Phase 2 Dose (RP2D) of AUTX-703

    To determine the RP2D of AUTX-703 based on safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity data.

    Measured: From the first dose through 28 days after the last dose of study drug.

  2. Peak Plasma Concentration (Cmax)

    Measured: From the first dose through the first treatment cycle (28 days)

  3. Time to Maximum Concentration (Tmax)

    Measured: From the first dose through the first treatment cycle (28 days)

  4. Area Under the Plasma Concentration-Time Curve from Time Zero to Infinity (AUCinf)

    Measured: From the first dose through the first treatment cycle (28 days)

  5. Area Under the Plasma Concentration-Time Curve to the Last Measurable Concentration (AUClast)

    Measured: From the first dose through the first treatment cycle (28 days)

  6. Elimination Half-Life (t½)

    Measured: From the first dose through the first treatment cycle (28 days)

  7. Apparent Clearance (CL/F)

    Measured: From the first dose through the first treatment cycle (28 days)

  8. Apparent Volume of Distribution (Vd/F)

    Measured: From the first dose through the first treatment cycle (28 days)

  9. To characterize the PD of AUTX-703

    To evaluate changes in KAT2A and KAT2B levels in peripheral blood and bone marrow as markers of pharmacodynamic response

    Measured: From the first dose through 28 days after the last dose of study drug

  10. AML: Complete remission (CR) rate

    Measured: Up to 18 months

  11. AML: CR + CRh rate

    Measured: Up to 18 months

  12. AML: Duration of CR

    Measured: Up to 18 months

  13. AML: Duration of CR + CRh

    Measured: Up to 18 months

  14. Objective response rate (ORR)

    Measured: Up to 24 months

  15. AML: Duration of response (DOR)

    Measured: Up to 18 months

  16. AML: Transfusion independence (TI) rate

    Measured: Up to 18 months

  17. AML: Event free survival (EFS)

    Measured: Up to 24 months

  18. AML: Overall survival (OS)

    Measured: Up to 24 months

  19. MDS: Complete remission (CR) rate

    Measured: Up to 18 months

  20. MDS: PR rate

    Measured: Up to 18 months

  21. MDS: CR+PR rate

    Measured: Up to 18 months

  22. MDS: Duration of CR

    Measured: Up to 18 months

  23. MDS: Duration of PR

    Measured: Up to 18 months

  24. MDS: Duration of CR+PR

    Measured: Up to 18 months

  25. MDS: Event free survival (EFS)

    Measured: Up to 24 months

  26. MDS: Overall survival (OS)

    Measured: Up to 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Feb 2025
  2. Study start1 May 2025
  3. Primary completionApr 2027 (estimated)
  4. Study completionJun 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
This study consists of two parts: Part A (Dose Escalation) and Part B (Dose Optimization). In Part A, participants are assigned sequentially to escalating dosages of AUTX-703 to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D). In Part B, participants are randomly assigned to receive one of two dosages of AUTX-703 for safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity.
Masking
None (open label)
Enrolment
69 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06846606
Sponsor's protocol ID
AUTX-703-001
Status last verified
Sep 2026
First submitted
6 Feb 2025
Last updated
16 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing AUTX-703 in people with Relapsed Acute Myeloid Leukemia (AML) and Refractory Acute Myeloid Leukemia (AML), run by Auron Therapeutics, Inc. at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Auron Clinical Trials (617-514-2356, clinicaltrials@aurontx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Adverse Events (AEs), Dose-Limiting Toxicities (DLTs), and Serious Adverse Events (SAEs) (time frame: From the first dose through 28 days after the last dose of study drug.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06846606.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.

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